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. 2020 Feb 7;5:8. doi: 10.1038/s41392-020-0110-5

Table 3.

Agents targeting CSC-associated signaling pathways and microenvironment in ongoing clinical trials

Drug name Target Condition Phase Sample size NCT number Current status
Hedgehog inhibitors
  Vismodegib (GDC-0449) Smoothened Recurrent or refractory medulloblastoma II 31 NCT00939484 Completed, has results
Basal cell carcinoma 28 NCT01700049 Completed, has results
Sarcoma 78 NCT01700049 Completed, has results
Recurrent small-cell lung carcinoma 168 NCT01700049 Completed, has results
Metastatic pancreatic cancer 98 NCT01088815 Completed, has results
Ovarian cancer 104 NCT00739661 Completed, has results
Metastatic colorectal cancer 199 NCT00636610 Completed, has results
  Sonidegib (LDE225) Basal cell carcinoma II 10 NCT01350115 Completed, has results
Relapsed medulloblastoma 20 NCT01708174 Completed, has results
Acute myeloid leukemia 70 NCT01826214 Completed, has results
Pancreatic adenocarcinoma 20 NCT01431794 Completed, has results
Advanced or metastatic hepatocellular carcinoma 9 NCT02151864 Completed
Recurrent plasma cell myeloma 28 NCT02086552 Active, not recruiting, has results
Advanced pancreatic cancer 39 NCT01485744 Active, not recruiting
Advanced breast cancer I 12 NCT02027376 Completed, has results
  Glasdegib Acute myeloid leukemia II 255 NCT01546038 Completed, has results
  BMS-833923 (XL139) Solid tumors II 12 NCT01413906 Completed
Small-cell lung carcinoma 5 NCT00927875 Completed
Metastatic gastric, gastroesophageal, esophageal adenocarcinomas 39 NCT00909402 Completed
Advanced or metastatic basal cell carcinoma 53 NCT00670189 Completed
Leukemia 70 NCT01357655 Terminated, has results
  Taladegib (LY2940680) Localized esophageal or gastroesophageal junction cancer II 9 NCT02530437 Active, not recruiting
Small-cell lung carcinoma 26 NCT01722292 Terminated, has results
  LEQ-506 Solid tumors I 57 NCT01106508 Completed
  G-024856 BCC I
  Patidegib (IPI-926) Basal cell carcinomas II 36 NCT02828111 Completed, has results
Metastatic or locally advanced chondrosarcoma 105 NCT01310816 Completed
Metastatic pancreatic cancer 122 NCT01130142 Completed
Recurrent head and neck cancer I 9 NCT01255800 Completed
Notch inhibitors
  MK-0752 γ-Secretase Advanced breast cancer I 103 NCT00106145 Completed
Pancreatic cancer I 44 NCT01098344 Completed
Metastatic breast cancer I/II 30 NCT00645333 Completed, has results
  RO4929097 Recurrent melanoma II 14 NCT01196416 Completed, has results
Advanced or metastatic sarcoma 78 NCT01154452 Completed, has results
Recurrent renal cell carcinoma 12 NCT01141569 Completed, has results
Advanced solid tumors 20 NCT01131234 Completed
Recurrent and/or metastatic epithelial ovarian cancer, fallopian tube cancer, or primary peritoneal cancer 45 NCT01175343 Completed, has results
Metastatic pancreas cancer 18 NCT01232829 Completed, has results
Recurrent colon cancer 37 NCT01116687 Completed, has results
Recurrent or refractory non-small-cell lung cancer 7 NCT01070927 Completed
  Nirogacestat (PF-03084014) Metastatic cancer pancreas II 3 NCT02109445 Terminated, has results
Fibromatosis II 17 NCT01981551 Active, not recruiting
Triple-negative breast neoplasms II 19 NCT02299635 Terminated, has results
  LY900009 Advanced cancer I 35 NCT01158404 Completed, has results
  Crenigacestat (LY3039478) Pan-Notch Advanced solid tumor I 12 NCT02836600 Active, not recruiting
T cell acute lymphoblastic leukemia, T cell lymphoblastic lymphoma I/II 36 NCT02518113 Completed, has results
  AL101 Adenoid cystic carcinoma II 36 NCT03691207 Recruiting
  CB-103 Advanced or metastatic solid tumors and hematological malignancies I/II 165 NCT03422679 Recruiting
  BMS-906024 Advanced or metastatic solid tumors I 94 NCT01292655 Completed
Lymphoblastic leukemia, acute T cell I 31 NCT01363817 Completed
  Demcizumab (OMP-21M18) DLL4 Pancreatic cancer II 207 NCT02289898 Completed, has results
Non-squamous, non-small-cell neoplasm of lung II 82 NCT02259582 Completed, has results
  Brontictuzumab (OMP-52M51) Adenoid cystic carcinoma Not applicable 1 NCT02662608 Completed, has results
  Enoticumab (MEDI528) Advanced solid malignancies I 83 NCT00871559 Completed
  MEDI0639 Solid tumors I 58 NCT01577745 Completed, has results
Wnt inhibitors
  Ipafricept (OMP-54F28) Wnt receptor Solid tumors I 26 NCT01608867 Completed
Pancreatic cancer I 26 NCT02050178 Completed
Ovarian cancer I 37 NCT02092363 Completed
Hepatocellular cancer I 10 NCT02069145 Completed
  Vantictumab (OMP-18R5) Metastatic breast cancer I 37 NCT01973309 Completed
Solid tumors I 35 NCT01345201 Completed
Pancreatic cancer I 30 NCT02005315 Completed
  PRI-724 β-Catenin/CBP Colorectal adenocarcinoma II 0 NCT02413853 Withdrawn
Acute myeloid leukemia 49 NCT01606579 Completed
Solid tumors 23 NCT01302405 Terminated
Advanced pancreatic cancer 20 NCT01764477 Completed
  CWP232291 Acute myeloid leukemia I 69 NCT01398462 Completed
Multiple myeloma I 25 NCT02426723 Completed
  LGK974 Porcupine Metastatic colorectal cancer I 20 NCT02278133 Completed
Pancreatic cancer I 170 NCT01351103 Recruiting
  ETC-1922159 Solid tumors I 65 NCT02521844 Active, not recruiting
Other signaling pathways inhibitors
  Galunisertib (LY2157299) TGF-β Prostate cancer II 60 NCT02452008 Recruiting
  LY3200882 Colorectal cancer II 31 NCT04031872 Not yet recruiting
  AVID200 Malignant solid tumor I 36 NCT03834662 Recruiting
  Trabedersen (AP 12009) Pancreatic neoplasms II 62 NCT00844064 Completed
Breast cancer 16 NCT01959490 Completed, has results
Glioblastoma 141 NCT00431561 Completed
  Fresolimumab (GC1008) Non-small-cell lung carcinoma II 60 NCT02581787 Recruiting
Metastatic Breast Cancer 23 NCT01401062 Completed, has results

Carcinoma

Renal cell

Melanoma

 29 NCT00356460 Completed
  Vactosertib (TEW-7197) Advanced-stage solid tumors I 35 NCT02160106 Completed
  NIS793

Breast cancer

Lung cancer

Hepatocellular cancer

 I 220 NCT02947165 Recruiting
  Ruxolitinib JAK Metastatic breast cancer III 29 NCT01594216 Completed
Myeloproliferative neoplasms 309 NCT00952289 Completed, has results
  AZD4205 Advanced non-small-cell lung cancer II 120 NCT03450330 Recruiting
  SAR302503 Hematopoietic neoplasm II 97 NCT01523171 Completed
  SB1518 JAK/FLT3 Acute myelogenous leukemia II 76 NCT00719836 Completed
PI3K inhibitors
  Alpelisib PI3K Advanced breast cancer II 90 NCT03386162 Recruiting
  Buparlisib (BKM120) Triple-negative metastatic breast cancer II 50 NCT01629615 Completed
  BYL719 Advanced or metastatic gastric cancer I 18 NCT01613950 Completed
  SF1126 Advanced or metastatic solid tumors I 44 NCT00907205 Completed
  SAR245409 PI3K and mTOR Advanced or metastatic solid tumors I 146 NCT01390818 Completed, has results
EGFR inhibitors
  Bevacizumab EGFR Breast cancer I 75 NCT01190345 Completed
  Matuzumab (EMD 72000) Esophageal cancer II 72 NCT00215644 Completed, has results
Non-small-cell lung carcinoma 150 NCT00111839 Completed, has results
Metabolism inhibitors
  Venetoclax (ABT-199) BCL-2 Acute myelogenous leukemia II 32 NCT01994837 Completed, has results
  Pegzilarginase Recombinant pegylated arginase Small-cell lung cancer II 84 NCT03371979 Active, not recruiting
  131I-TLX-101 LAT1 Glioblastoma multiforme II 44 NCT03849105 Recruiting
  Rifampicin FAS Advanced solid tumors I 36 NCT03077607 Completed, has results
  TVB-2640 Advanced breast cancer II 80 NCT03179904 Recruiting
  IM156 AMPK Advanced solid tumor I 36 NCT03272256 Recruiting
  Telaglenastat Glutaminase Solid tumors II 85 NCT03965845 Recruiting
  CB-1158 Arginase Advanced solid tumors II 5 NCT03361228 Completed
Niche inhibitors
  Plerixafor (Mozobil) CXCR4 Advanced pancreatic, ovarian, and colorectal cancers I 26 NCT02179970 Completed
  BL-8040 Metastatic pancreatic adenocarcinoma II 23 NCT02907099 Active, not recruiting
  BKT140 Multiple myeloma II 16 NCT01010880 Completed
  BMS-936564 Relapsed/refractory multiple myeloma I 46 NCT01359657 Completed
  BMS-936564 Acute myelogenous leukemia I 98 NCT01120457 Completed
  LY2510924 Solid tumor I 9 NCT02737072 Terminated, has results
  MSX-122 Refractory metastatic or locally advanced solid tumors I 27 NCT00591682 Suspended
  USL311 Advanced solid tumors and relapsed/recurrent Glioblastoma multiforme II 120 NCT02765165 Recruiting
  AMD3100 Acute myeloid leukemia II 52 NCT00512252 Completed, has results
  Reparixin CXCR1/2 Breast cancer II 20 NCT01861054 Terminated
  Defactinib (VS-6063) FAK Non-small-cell lung cancer II 55 NCT01951690 Completed